Very titillating: Untested – designer cell – therapy saves a baby close to dying from leukemia – The Washington Post

Baby Layla is seen at Excellent Ormond Street Hospital (GOSH) in London in this October 28, two thousand fifteen handout photo by the hospital released on November Five, 2015. (Sharon Lees/Fine Ormond Street Hospital/Reuters)

Five months ago, Layla Richards’s parents were told that their infant daughter was about to die.

The child had been undergoing treatment for acute lymphoblastic leukemia since she was just fourteen weeks old. She’d been subjected to chemotherapy, flown across the U.K. for experimental procedures, undergone bone marrow transplants and suffered uncountable blood tests. The majority of her year-long life had been spent in intensive care units. And still, cancer seemed to have conquered her petite figure.

“Doctors don’t want to say that ‘there’s nothing we can do’ and suggest palliative care, but sometimes that’s the only option,” Layla’s mother, Lisa Foley, said in a hospital press release.

But the little girl’s family took the only option and rejected that too. “We didn’t want to … give up on our daughter,” Foley continued. “So we asked the doctors to attempt anything.”

“Anything” turned out to be a very experimental and possibly ineffective therapy that had never before been tested on anything larger than a lab mouse. It might not have any effect on Layla’s leukemia, the doctors warned. It might even make her sicker.

Do it, her parents said. “We had to do something.”

That’s how Layla Richards, a 1-year-old from London, became the very first person in the world to be treated for cancer using “designer immune cells,” according to a write-up in “Nature,” which noted that the method has been attempted on patients with HIV. If her bod remains cancer-free, as it has been for the past several months, she could become the very first person to be cured by them. According to Nature, the team will present the case in December at a meeting of the American Society of Hematology.

The medical team cautioned that the therapy will require much more testing and has a long way to go before it could be approved for broader usage.

“It’s very titillating but it is early days,” Matt Kaiser, head of research at the British charity Bloodwise, which funds research on blood cancers, told the Wall Street Journal. Kaiser wasn’t involved in Layla’s treatment. To validate the treatment attempted in Layla, he continued, “there clearly needs to be long term clinical studies, with more patients. We need to understand who responds and who doesn’t, and what the long term results are.”

The treatment, which involves using lil’ molecular “scissors” to edit genes and make immune cells especially capable of hunting out and ruining cancer, represents a promising fresh front in the fight against cancer. If it works — not just in Layla, but in future lab tests and clinical trials — doctors might one day be able to pluck a vial of genetically engineered immune cells from a shelf and then inject them into a patient to wage microscopic war against previously unresponsive cancers.

A similar treatment, known as CAR T-cell therapy (T-cells are the immune cells that fight off cellular abnormalities and infections, CAR is the protein that gets added to T-cells that equips them to kill cancer) has been successful in petite trials for patients whose leukemia didn’t react to chemotherapy. But as the Fresh York Times pointed out, those treatments require that immune cells be extracted from a patient, shipped to a plant to be altered, sent back and reinjected into the patient — a process that can be lengthy, logistically challenging and expensive. In addition, patients who have already undergone several rounds of excruciating and wearisome chemotherapy may not have enough healthy T-cells left for this course of treatment.

The therapy used to treat Layla, which was developed by researchers at Superb Ormond Street Hospital (GOSH) and University College London’s (UCL) Institute of Children’s Health along with the biotech company Cellectis, takes another tack.

Instead of relying on the patient’s weakened immune cells, the researchers developed a bank of pre-engineered, one-size-fits-all T-cells from healthy donors. Using a genome editing instrument called TALEN, they cut the T-cells to render them impervious to leukemia drugs, which would ordinarily kill them, and paste in fresh programming that directs them to hunt down and fight against cancer. The editing also prevents the donor cells from attacking anything else it comes across (like the patient’s own cells) a concern whenever someone is injected with cells that aren’t theirs.

These genetically engineered “designer cells” can be bottled up in vials and shipped to doctors, who then inject them into patients suffering from certain severe cancers — no extra assembly required.

Baby Layla (L) is seen with her parents, Lisa and Ashleigh, and her older sister Reya at Superb Ormond Street Hospital (GOSH) in London in this November Four, two thousand fifteen handout photo by the hospital released on November Five, 2015. (Fine Ormond Street Hospital/Reuters)

The very first “off the shelf” banks of donor T-cells, called UCART19, were still being tested when Layla’s parents asked if there was anything else the doctors at GOSH could do for their daughter, according to a hospital press release.

A consultant immunologist at GOSH who had been working on the therapy, Waseem Qasim of UCL, suggested UCART19. The idea was approved by an ethical panel, and a brief while later Layla was injected with a one milliliter vial of the cancer-fighting cells.

“We thought that the little bit of liquid in the injection needle was nothing,” Ashleigh Richards recalled in the press release. He and his wifey asked, “What is that going to do when bags and bags of chemo haven’t worked?”

The nurse reassured them. This kind of treatment was about quality, not quantity.

For two weeks, the family waited, and waited, and waited, for something to happen. Layla didn’t seem to be getting sicker. But she wasn’t getting better either. For a while, doctors thought about sending Layla home, telling her family to spend what time she had left together, beyond the beeping monitors and sterile white walls.

Then “the rash” appeared.

The rash was a telltale sign that her assets recognized and was responding to the infusion of foreign cells. A few weeks later, Foley was picking up the couple’s older daughter from school when her hubby called. Sit down, he told her.

“I thought it was bad news but then he said, ‘It worked,’ and I just cried blessed tears,” Foley said.

Her cancer gone, Layla underwent an operation to substitute the bone marrow that had deteriorated during treatment. She’s not “cured” yet — that won’t be certain for months or even years — but she’s well enough to go home, the hospital announced Thursday.

“Her leukemia was so aggressive that such a response is almost a miracle,” Paul Veys, Layla’s lead doctor, told Reuters.

Qasim, the UCL researcher, cautioned that UCART19 therapy still has a long way to go before it becomes a treatment option: “We have only used this treatment on one very strong little chick,” he said in the hospital’s press release. “… But this is a landmark in the use of fresh gene engineering technology and the effects for this child have been staggering.”

“If replicated, it could represent a gigantic step forward in treating leukemia and other cancers.”